WP44714 is a Phase I/II, open-label, non-randomized, global, multicenter trial consisting of two parts:
* Part 1 is a multiple-ascending dose (MAD) study in adult and adolescent male participants with severe or moderate hemophilia A with or without factor VIII (FVIII) inhibitors. * Part 2 is a multiple-dose study in pediatric male participants with severe or moderate hemophilia A with or without FVIII inhibitors.
The overall aim of the study is to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of NXT007.
Diagnosis of severe (Factor VIII coagulant activity [FVIII:C] <1 IU/dL) or moderate (FVIII:C ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A with or without inhibitors against FVIII
Participants with FVIII inhibitors: participants using recombinant activated factor VII (rFVIIa) or willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds, trauma, or procedures
Historic local FVIII inhibitor test results being available during screening to confirm any previous inhibitor history and current status
Participants who previously successfully completed immune tolerance induction (ITI) must have done so at least 5 years before screening and must have no evidence of inhibitor recurrence (permanent or temporary) since. FVIII tolerance defined as <0.6 Bethesda unit (BU)/mL (<1.0 BU/mL only for laboratories with an historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and in vivo recovery >66%
Inherited or acquired bleeding disorders other than congenital hemophilia A
Ongoing or planned ITI therapy
Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
At high risk for thrombotic microangiopathy (TMA), including past personal or family history of TMA, in the investigator's judgment
Sequential
NXT007
DrugParticipants will receive NXT007 administered subcutaneously (SC), 2 loading doses once every two weeks (Q2W) followed by once every 4 weeks (Q4W) maintenance doses based on the schedule.
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Treatment groupsSee each treatment group below.
Incidence and Severity of Adverse Events, with Severity Determined According to National Cancer Institute Common Terminology Criteria for Adverse Events Grading Scale
Number of Participants with at Least One Clinical Laboratory Test Abnormality for Hematology Parameters
Number of Participants with at Least One Clinical Laboratory Test Abnormality for Blood Chemistry Parameters
Number of Participants with at Least One Vital Sign Abnormality
The vital signs that will be assessed are body temperature, pulse rate, respiratory rate, and systolic and diastolic blood pressure.
Plasma Concentration of NXT007 at Specified Timepoints
Maximum Observed Plasma Concentration (Cmax) of NXT007 After the First Dose
Time to Maximum Observed Plasma Concentration (tmax) of NXT007 After the First Dose
Area Under the Plasma Concentration-Time Curve (AUC) of NXT007 After the First Dose
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